A Retrospective, Natural History Study in Children With CLN2

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Study Classification

OBSERVATIONAL

Study Start Date

2021-03-31

Study Completion Date

2022-04-30

Brief Summary

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This is a multi center, retrospective, chart review study to document the evolution of ocular disease progression in pediatric patients with CLN2.

Detailed Description

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CLN2 is a rare disease with limited available ocular natural history data. While current standard of care slows motor degeneration, it is not known to treat the ocular manifestations of disease. This study is planned to document, through retrospective data collection, ocular disease progression in children with a clinical presentation consistent with CLN2. No investigational product is administered in this retrospective, chart review study.

Conditions

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Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)

Keywords

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CLN2, Batten Disease

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

RETROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

A participant is eligible to be included in the study only if all of the following criteria apply:

1. The participant's legal guardian(s) is(are) willing and able to provide them written, signed informed consent.
2. The participant has a documented diagnosis of CLN2 disease due to TPP1 deficiency, or has a relative clinically diagnosed with CLN2 disease who has the same CLN2 mutations as the participant
3. The participant has had one or more eye examinations by an eye care specialist at any time since birth.

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Other Identifiers

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RGX-381-9102

Identifier Type: -

Identifier Source: org_study_id